A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4
This is a multicenter, global study of the effects of a single systemic dose of SRP-9003 on beta-sarcoglycan (β-SG) gene expression in participants with limb-girdle muscular dystrophy, type 2E/R4 (LGMD2E/R4). This study will consist of both ambulatory participants (Cohort 1) and non-ambulatory participants (Cohort 2).
Eligibility
Sex
ALL
Min age
4 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria:
* Cohort 1, only ambulatory participants:
* Able to walk without assistive aid
* 10MWR \<30 seconds
* NSAD ≥25
* Cohort 2, only non-ambulatory participants:
* 10MWR ≥30 seconds or unable to perform
* PUL 2.0 entry scale score ≥3
* Participants must possess 1 homozygous or 2 heterozygous pathogenic and/or likely pathogenic β-SG DNA gene mutations
* Able to cooperate with muscle testing
* Participants must have adeno-associated virus serotype rh74 (AAVrh74) antibody titers \<1:400 (that is, not elevated) as determined by AAVrh74 antibody enzyme-linked immunosorbent assay.
Exclusion Criteria:
* Left ventricular ejection fraction \< 40% or clinical signs and/or symptoms of cardiomyopathy
* Forced vital capacity ≤40% of predicted value and/or requirement for nocturnal ventilation
* Diagnosis of (or ongoing treatment for) an autoimmune disease and on active immunosuppressant treatment
* Presence of any other clinically significant illness or medical condition (other than LGMD2E/R4)
Other inclusion/exclusion criteria apply.
Primary outcome measure(s)
Cohort 1: Change from Baseline in β-SG Expression at Day 60 Post-dose as Measured by Immunofluorescence (IF) Percent β-SG Positive Fibers — Baseline, Day 60
Trial sites (10)
Facility
City
Region
Status
University of California, San Diego-Altman Clinical and Translational Research Institute
La Jolla
California
Nationwide Childrens Hospital
Columbus
Ohio
The Children's Hospital of Philadelphia
Philadelphia
Pennsylvania
Children's Hospital of The King's Daughter
Norfolk
Virginia
University Hospital Leuven (UZ Leuven)
Leuven
Vlaams Brabant
NMRC Gent (UZ Gent)
Ghent
Belgium
Universitatsklinikum Essen; Kinderklinik I, Sozialpadiatrisches Zentrum
Essen
North Rhine-Westphalia
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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